Septerna (SEPN) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Pipeline overview and strategic focus

• Focused on GPCR drug discovery, enabling novel compounds for challenging targets, including small molecule agonists and allosteric modulators.

• Lead program SEP-479 is in phase I for hypoparathyroidism, with additional programs targeting mast cell diseases, thyroid disorders, and osteoporosis.

• Novo Nordisk partnership targets metabolic diseases, leveraging novel binding pockets in incretin receptors.

• Strong financial position with $516 million in cash, providing runway into 2029.

SEP-479 (hypoparathyroidism) development

• SEP-479 designed to mimic PTH peptide, showing promising translation from animal models to healthy volunteers.

• Phase I study uses single and multiple ascending dose design, with a long half-life (~3-4 days) considered optimal.

• Plans to rapidly transition to global phase II, including open-label extension and chronic toxicology studies.

• Phase II will test starting doses and titration protocols, leveraging learnings from Yorvipath.

• Pivotal studies expected to be around 100 patients, with potential for combining phase II and III data.

Commercial and competitive landscape for SEP-479

• Oral therapy aims to address unmet needs for patients not well-controlled on current standard of care or unwilling to use injectables.

• Switch studies planned to facilitate transition from injectable to oral therapy.

• Ex-U.S. commercialization strategy includes comprehensive dossiers to support pricing and access globally.

• Product expected to appeal to both severe and mild patients, potentially expanding the treatable population.